Abstract

A trial drug encourages cells to ignore the signs that stop them making faulty proteins. Sound dangerous? Claire Ainsworth discovers that it could be a cure for genetic disease. Nonsense-mediated decay (NMD) was first identified as a pathway that prevents abnormal protein synthesis by eliminating mRNAs with premature stop codons. It's now clear that NMD has a role in controlling some genetic diseases, but that it can have a harmful effect in others. A difficult balancing act for those working on NMD as a possible means of therapy.

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